Biotechnology company Sensorion (Paris: ALSEN) on Wednesday announced encouraging early results from its Audiogene Phase 1/2 clinical trial evaluating SENS-501 (OTOF-GT), a gene therapy targeting congenital hearing loss caused by otoferlin (OTOF) gene mutations. The first two toddlers treated, aged 6 to 31 months, demonstrated no serious adverse events and showed promising behavioural improvements.

The trial's primary goal in its dose-escalation phase is to assess the safety of SENS-501 delivered via intra-cochlear injection. Targeting infants and toddlers leverages optimal brain plasticity to maximize potential for speech and language development. By introducing a functional OTOF gene using AAV viral vector technology, the therapy aims to restore auditory signal transmission, addressing severe to profound genetic hearing loss.

Sensorion plans to complete the first cohort by year-end and begin enrolling a second cohort by mid-2025. A KOL event is scheduled for early 2025 to review safety data, discuss efficacy metrics and outline next steps, including FDA engagement.

SENS-501 is part of Sensorion's broader gene therapy pipeline, developed in collaboration with Institut Pasteur and supported by RHU AUDINNOVE. Alongside SENS-401, a Phase 2 small molecule candidate, the company aims to address critical unmet needs in hearing loss treatment and prevention.